A mutant granule-bound starch synthase 1 (GBSS1) polypeptide and a nucleic acid, and use thereof are provided. Compared to an amino acid sequence of a parent GBSS1, the mutant GBSS1 polypeptide has a mutation at an amino acid corresponding to amino acid 427 and/or amino acid 428 of an amino acid sequence shown in SEQ ID NO: 1. An amylose content (AC) in a plant changes after the plant undergoes GBSS1 mutation, which has very promising application prospects in the improvement of edible quality of rice.

This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.

A method for treating a neurodegenerative disease is provided. The method for treating a neurodegenerative disease includes administering, to a subject in need thereof, one or more of a small molecule glycogen synthase (GYS) inhibitor, an antisense oligonucleotide targeting glycogen synthase, an antibody-enzyme fusion compound targeting polyglucosan bodies (PGBs), or combinations thereof.

A mutant granule-bound starch synthase 1 (GBSS1) polypeptide and a nucleic acid, and use thereof are provided. Compared to an amino acid sequence of a parent GBSS1, the mutant GBSS1 polypeptide has a mutation at an amino acid corresponding to amino acid 427 and/or amino acid 428 of an amino acid sequence shown in SEQ ID NO: 1. An amylose content (AC) in a plant changes after the plant undergoes GBSS1 mutation, which has very promising application prospects in the improvement of edible quality of rice.

The present disclosure provides methanotrophic bacteria that are modified to produce less glycogen, and methods of using the modified methanotrophic bacteria to produce a desired product, such as protein(s) or metabolite(s).

This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.

The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

A mutant granule-bound starch synthase 1 (GBSS1) polypeptide and a nucleic acid, and use thereof are provided. Compared to an amino acid sequence of a parent GBSS1, the mutant GBSS1 polypeptide has a mutation at an amino acid corresponding to one or more of amino acid 237, amino acid 168, and amino acid 411 of an amino acid sequence shown in SEQ ID NO: 1. An amylose content (AC) in a plant changes after the plant undergoes GBSS1 mutation, which has very promising application prospects in the improvement of edible quality of rice.

A mutant granule-bound starch synthase 1 (GBSS1) polypeptide and a nucleic acid, and use thereof are provided. Compared to an amino acid sequence of a parent GBSS1, the mutant GBSS1 polypeptide has a mutation at an amino acid corresponding to one or more of amino acid 237, amino acid 168, and amino acid 411 of an amino acid sequence shown in SEQ ID NO: 1. An amylose content (AC) in a plant changes after the plant undergoes GBSS1 mutation, which has very promising application prospects in the improvement of edible quality of rice.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload reduces glycogen levels in a cell.